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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05187260
Other study ID # MRCTA,ECFAH of FMU [2021]489
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date January 1, 2022
Est. completion date January 31, 2024

Study information

Verified date January 2022
Source First Affiliated Hospital of Fujian Medical University
Contact Yi Lin, MD, PhD
Phone 13615039153
Email linyi7811@163.com
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 months.


Description:

SPINRAZA® (nusinersen) is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Nusinersen, approved by the FDA for treatment of SMA in 2016, was approved by the Chinese National Medical Products Administration in 2019. This is a prospective, longitudinal, multi-center, observational study designed to evaluate the efficacy, safety, tolerability and of nusinersen in patients genetically confirmed chromosome 5q SMA in China. Subjects with SMA I/II/III who are planning to initiate treatment with nusinersen will be enrolled in this study. All patients will be treated by their physicians according to standard clinical practice. SPINRAZA® (nusinersen) is administered as an intrathecal injection. A total of 5ml of cerebrospinal fluid (CSF) will be removed prior to administration of SPINRAZA® (nusinersen), which will be collected by the study. Neurofilament light chain (NfL) in CSF and blood will be assessed for the efficacy of nusinersen, as well as motor and pulmonary function. There will be a total of nine visits. All the patients with 5q SMA receiving nusinersen will be visited face to face at baseline, day 14, day 28 and day 63 after treatment initiation, and then 4-month intervals through month 24/22.


Recruitment information / eligibility

Status Recruiting
Enrollment 1000
Est. completion date January 31, 2024
Est. primary completion date January 31, 2024
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group N/A to 50 Years
Eligibility Inclusion Criteria: - Patients genetically confirmed 5q SMA including types I, II and III, who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan. - Non-5q SMA patients undergoing clinical standard lumbar puncture - Non-SMA subjects including Asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture - Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process Exclusion Criteria: - Contraindication for lumbar puncture - Inability to access intrathecal space for nusinersen injection

Study Design


Intervention

Drug:
Nusinersen
SPINRAZA® (nusinersen) prescribed as part of standard of care

Locations

Country Name City State
China Department of Neurology, Peking Union Medical College Hospital Beijing Beijing
China Department of Neurology, First Affiliated Hospital Fujian Medical University Fuzhou Fujian
China Department of Pediatrics, Fujian Medical University Union Hospital Fuzhou Fujian

Sponsors (3)

Lead Sponsor Collaborator
First Affiliated Hospital of Fujian Medical University Fujian Medical University Union Hospital, Peking Union Medical College Hospital

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change in CSF Neurofilament Light Chain levels since baseline Measured by Single-molecule Array up to 24months
Secondary Change in serum Neurofilament Light Chain levels since baseline Measured by Single-molecule Array up to 24months
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